EU/3/09/668 - orphan designation for treatment of angioedema caused by C1 inhibitor deficiency

human C1 inhibitor

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2011 upon request of the marketing authorisation holder at the time of the granting of a marketing authorisation.

On 8 October 2009, orphan designation (EU/3/09/668) was granted by the European Commission to ViroPharma SPRL, Belgium, for human C1 inhibitor for the treatment of angioedema caused by C1 inhibitor deficiency.

What is angioedema caused by C1 inhibitor deficiency?

Angioedema is a disease characterised by attacks of swelling beneath the skin that can occur anywhere in the body, such as in the face, limbs, gut and larynx (voice box), causing discomfort and pain.

Angioedema can be caused by the deficiency (low levels) of 'C1 inhibitor', a protein in the blood that prevents the activation of some proteins involved in causing inflammation (swelling). The C1 inhibitor deficiency can be 'hereditary' or 'acquired'. Hereditary angioedema is caused by abnormalities in the gene responsible for the production of C1 inhibitor. Acquired angioedema is caused by conditions that increase the breakdown of C1 inhibitor such as cancer of the B cells (a type of white blood cell) and diseases where the body's own defence system attacks the C1 inhibitor protein.

Angioedema caused by C1 inhibitor deficiency is a long-term debilitating disease that may be life threatening because, when the swelling occurs in the larynx, it can obstruct the airways and impede breathing.

What is the estimated number of patients affected by the condition?

At the time of designation, angioedema caused by C1 inhibitor deficiency affected approximately 2.1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 106,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for the treatment of angioedema. These included medicines containing human C1 inhibitor, which were authorised in some EU countries for the treatment and prevention of angioedema, and icatibant, which was authorised in all EU countries to treat attacks of hereditary angioedema.

The sponsor has provided sufficient information to show that human C1 inhibitor might be of significant benefit for patients with angioedema caused by C1 inhibitor deficiency because it may offer a wider availability than some of existing treatments, which could result in a major contribution to patient care. In addition, the properties of the product, with regards to its duration of action, might offer a clinically relevant advantage over some authorised products. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

Human C1 inhibitor is extracted from human plasma (the liquid part of the blood) obtained from blood donors and is expected to work by increasing the levels of the C1 inhibitor protein in patients with angioedema. The C1 inhibitor protein is then expected to prevent the activation of proteins in the blood that are involved in inflammation. By inhibiting these proteins, human C1 inhibitor may reduce the symptoms of angioedema caused by C1 inhibitor deficiency.

What is the stage of development of this medicine?

The effects of human C1 inhibitor have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with angioedema caused by C1 inhibitor deficiency were ongoing.

At the time of submission, this human C1 inhibitor was not authorised anywhere in the EU for angioedema caused by C1 inhibitor deficiency. Orphan designation of human C1 inhibitor had been granted in the United States of America for routine prophylaxis (preventative treatment) of hereditary angioedema.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 July 2009 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the European Union) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

EU/3/09/668: Public summary of positive opinion for orphan designation of human C1 inhibitor for the treatment of angioedema caused by C1 inhibitor deficiency

Adopted Reference Number: EMEA/COMP/470276/2009

Key facts

• Active substance:
  human C1 inhibitor
• Intended use:
  Treatment of angioedema caused by C1 inhibitor deficiency
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/09/668
• Date of designation:
  08/10/2009
• Sponsor:

  ViroPharma SPRL; Square de Meeûs 37; 1000 Brussels; Belgium; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]